Improving Outcomes with Haploidentical Stem Cell Transplantation [HaploSCT] in Children Using Post-transplant
Biju George1, Uday Kulkarni1, Sharon Lionel1
1Department of Haematology, Christian Medical College, Vellore, India.
Summary
Haplo-identical stem cell transplants are a viable option for children lacking a matched sibling donor, offering improved survival rates over time. Further research is needed to reduce graft failure and infections, which negatively impact outcomes.
Area of Science:
- Pediatric Hematology
- Transplant Immunology
- Oncohematology
Background:
- Haplo-identical stem cell transplantation (SCT) with post-transplant cyclophosphamide is an emerging alternative for pediatric patients without a matched sibling donor.
- This approach is increasingly utilized for both malignant and non-malignant pediatric disorders.
Purpose of the Study:
- To evaluate the outcomes of haplo-identical SCT using post-transplant cyclophosphamide in children.
- To identify factors influencing overall survival in this patient population.
Main Methods:
- Retrospective analysis of 138 SCT procedures in 127 children between 2010 and June 2021.
- Utilized both myeloablative and reduced intensity conditioning regimens with peripheral blood stem cells.
- Assessed engraftment, graft-versus-host disease (GVHD), infections, and overall survival (OS).
Main Results:
- Engraftment was achieved in 81.9% of patients, with a 10.2% primary graft failure rate.
- Cumulative incidence of acute and chronic GVHD was 49.5% and 40.7%, respectively.
- Two-year OS was 54.9%, with significantly lower survival in younger children (0-5 years). Survival rates improved over time (2010-2021).
- Bacterial infection, invasive fungal disease, and graft failure were identified as negative prognostic factors for OS.
Conclusions:
- Haplo-identical SCT with post-transplant cyclophosphamide is a feasible option for pediatric patients lacking a matched sibling donor.
- Strategies to mitigate graft failure, infection-related mortality, and GVHD are crucial for improving long-term outcomes.
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